THINGS

YOU NEED TO

KNOW ABOUT PATIENT REPORTED OUTCOME

(PRO) MEASURES

Introduction

With the increasing prominence of the patient’s involvement in the care

they receive, the assessment of outcomes based on the patient’s perspective using patient reported outcome

measures (PROMs), are increasingly accompanying the traditional clinical ways of measuring health and the

effects of treatment on the patient. However, with literally scores of PROMs to choose from you need to do more

than base your selection on the name of the PROM, what it claims to measure or because it’s been used by

others. You need to be assured that it’s appropriate for measuring the desired outcomes. You need evidence of

the PROMs reliability and validity and above all you need to have a clearly defined measurement strategy that

links the outcomes with the disease and treatment outcomes.

Selecting the most appropriate PROM is more than assuming the title of the PROM will

tell you all you need to know or that the PROM has been used in numerous studies.

Failure to consider key factors in the choice process will lead to the use of costly resource

in the collection of invalid and unreliable information.

This presentation provides you with some of the key factors in the decision making process when selecting an

appropriate PROM.

1: PROMs are the tools we use to gain insight from the perspective of the

patient into how aspects of their health and the impact the disease and

its treatment are perceived to be having on their lifestyle and

subsequently their quality of life (QoL). They are typically self-

completed questionnaires, which can be completed by a patient or

individual about themselves, or by others on their behalf.

A PROM should be designed to provide information around a given concept

which must be made explicit by the instruments’ authors. Common concepts

include:

What is a PROM & what does it measure?

Access to the patients’ perspective through the use of

PROMs can impact on a wide range of aspects related

to the delivery of effective health care including,

identifying those issues faced by patients and their

families living with an illness and how this knowledge

might impact on treatment.

EXAMPLE

Health status - Measures of health-status focus on the quality of health including, the

biological and physiological dysfunctioning, symptoms as well as the physical e.g. the ability to

climb a flight of stairs, as well as the psychological and social functional impairments.

Quality of life (QoL) - In defining QoL there is the general consensus that it is based on the

individual’s subjective evaluation of the psychological, physical and social aspects of their life,

which is changing over time as a result of different influences such as treatment5. QoL is what the

patient says it is.

Health related quality of life (HRQoL) - Often referred to as the degree to which the

treatment and the disease as perceived by the individual to impact on those aspects of their life -

in addition to health – which are considered important.

2: Selecting the most appropriate PROM is of course the most critical

aspect of the study design and in the absence of some universally

agreed definition as to what the measured health concept is and its

relationship to the objectives of the study, choosing the appropriate

PROM can be problematic.

Developing a measurement strategy An effective way to establish the link between the measured outcome such

as the patient’s well-being following an intervention programme is to

develop a measurement strategy, which requires a clear understanding of

the disease and the outcomes relevant to the disease area and patient. That

will help you select the appropriate PROM.

Selecting the appropriate PROM?

Identify key treatment effects and key outcomes

Select outcomes relevant to the treatment or intervention

Develop endpoint model

Select Patient Reported Outcome Measure (PROM)

It is not uncommon that the choice of an

outcome measure such is based on the

instrument been used in previous studies.

NOTE

3: What you need to know about reliability & validity

A PROM is only of value if it has been designed to strict

criteria and based on a sound theoretical underpinning or

conceptual model of what it is purported to measure .

“Reliability tells how consistent our results are, whilst validity

tell us whether we are measuring what we think we are”.

“Validity is telling us how well we are measuring what we

think we are measuring”.

4: When to apply a PROM

Data derived from a PROM can guide clinicians in making decisions

about different clinical inputs and for monitoring the outcomes of

specific interventions.

PROMs can also provide a baseline assessment of the health status, QoL,

patient satisfaction or well-being etc. of a specific population to identify

need and the delivery of effective care.

Aid communication between patient and doctor. For example, the doctor

can discuss with the patient why a question was answered in a particular

way or why the patient’s score has improved or declined since the

previous visit.

PROMs can also be routinely administered in clinical settings for audit and

quality assurance such as in the assessment of the effectiveness of

different procedures.

5: Interpretation of data derived from a PROM can be challenging, particularly

with regard to understanding the meaning of what a change or difference in

a score means clinically.

Interpretation of PROM data is linked with both the objectives of the study and the

constructs measured by the PROM and as a consequence should be a key factor in

the development of any PROM:

• Minimal Important Difference (MID) - the smallest difference in a score that is

considered to be worthwhile or important.

• Known groups – the mean scores underlying particular clinical groups or

clinical indicators which give rise to them and which can be used as a clinically

based benchmark to compare other groups.

• Normative and reference groups – mean scores from defined large populations

to provide normative data – typical scores – called norms. Mean scores from a

particular study can be compared with the population norms.

• Statistical significance – The statistical significance of the probability of

treatment (A) is better than treatment (B).

• Effect size – A way of quantifying the difference between two groups of

patients that has many advantages over the use of statistical significance

alone and emphasises the size of the difference rather than confounding this

with sample size.

• Cumulative distribution functions (CDF) - The CDF shows a continuous plot of

the proportion of patients at each point along the continuum of the scale

score continuum experiencing change at that level or lower levels.

Ways to interpret PROM data

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